Through our Center for Cancer & Blood Disorders (CCBD), we collaborate with universities, pharmaceutical sponsors, foundations and consortiums to bring groundbreaking research opportunities to families.
Hematology Research
Hematology research at Phoenix Children’s is advancing our understanding of rare bleeding and blood disorders, such as hemophilia, Von Willebrand disease, sickle cell disease, thalassemia, PKD, ITP, chronic neutropenia, thrombosis and severe aplastic anemia. Our research drives cutting-edge clinical care, offering patients and families access to innovative treatments.
The mission of our Clinical Research Program is to deliver the most effective treatments available today while pioneering new therapies to ensure we can cure every patient in the future.
Additionally, we partner with the Primary Immune Deficiency Treatment Consortium (PIDTC), a collaborative network of medical centers focused on advancing research, treatment and outcomes for patients with rare, life-threatening primary immune deficiency disorders through clinical trials and multidisciplinary collaboration.
Sickle Cell Disease Research
Sickle cell disease (SCD) is an inherited monogenic disorder marked by chronic anemia, unpredictable pain episodes and multisystem organ damage, leading to significant lifelong morbidity and early mortality. It is one of the most common chronic childhood illnesses and the most prevalent inherited hemolytic anemia. SCD affects approximately 100,000 individuals in the United States. At Phoenix Children’s, our hematologists are dedicated to advancing the prevention and treatment of SCD through clinical trials, registries and research grants.
Maa-Ohui Quarmyne, MD and the CCBD team at Phoenix Children’s participate in a CDC-funded Arizona Sickle Cell Data Collection (SCDC) program. This initiative gathers health information from individuals with sickle cell disease to analyze long-term trends in diagnosis, treatment and healthcare access across the United States. Arizona is one of the newest states to join the 16 states participating in this program.
The primary goal of this surveillance effort is to gain a comprehensive understanding of SCD at the population level in Arizona. By studying the epidemiology, healthcare utilization and health outcomes of SCD patients, we aim to drive meaningful policy changes that will enhance access to care and improve quality of life. Phoenix Children’s leads this program. The Center for Health Information and Research (CHIR) at ASU serves as the data hub. The findings from this data collection will help shape future policies to improve care and outcomes for individuals with sickle cell disease in Arizona.
Blood & Marrow Transplant Research
In collaboration with Mayo Clinic Arizona, the Ottosen Family Blood and Marrow Transplant Program, utilizes stem cell transplants to restore healthy cells for patients with various hematologic, oncologic and metabolic/genetic conditions. As the only freestanding children's hospital in Arizona with a dedicated bone marrow transplant program since 2003, Phoenix Children’s Center for Cancer & Blood Disorders provides comprehensive care throughout the transplant process and beyond. We achieve survival rates in the top 10% nationally with over 50 transplants annually and cellular therapies CAR-T cell treatment.
Oncology Research
Over the past 30 years, the survival rate for children with cancer has significantly improved, thanks to several key factors:
- The development of new, more effective and targeted drugs and treatment regimens
- Advanced techniques like bone marrow transplantation and cellular therapies
- Innovative diagnostic and genetic testing, as well as imaging and surgical techniques
- Enhancements in supportive care measure
However, the most impactful factor in the fight against childhood cancer has been the widespread participation of pediatric cancer patients in clinical trials. These trials combine cutting-edge treatments with patient-focused research to evaluate new therapies.
Phoenix Children’s is deeply committed to advancing cancer treatment through clinical trials. By participating in national and international trials, we aim to identify more effective therapies for children with cancer. Expanding the number of children enrolled in well-designed studies will accelerate the evaluation and approval of promising new treatments.
Clinical Research Studies
Our team includes nationally recognized leaders with expertise in managing and developing innovative treatments for a wide range of pediatric cancers. The comprehensive Clinical Research Program at the CCBD offers a broad spectrum of clinical trials, providing patients with access to progressive therapies tailored to their specific cancer types, such as brain tumors, leukemias and lymphomas and solid tumors, including bone tumors, sarcomas and neuroblastoma.
Research Consortiums and Initiatives
Phoenix Children’s plays an active role in several national and international research groups that aim to advance pediatric cancer care and treatment. The hospital collaborates with Children's Oncology Group (COG), the world’s largest organization dedicated to pediatric cancer research, ensuring patients have access to the latest clinical trials. Through its partnership with the Pediatric Oncology Experimental Therapeutics Investigators' Consortium (POETIC), Phoenix Children’s contributes to developing experimental therapies for high-risk pediatric cancers.
The Pediatric Transplantation & Cellular Therapy Consortium (PTCTC) further enhances Phoenix Children’s efforts in cellular therapies and transplantation. The Beat Childhood Cancer Research Consortium (BCC) initiative works toward discovering targeted therapies for childhood cancers. These alliances are vital to offering advanced, innovative treatments for pediatric patients.
Phoenix Children’s pediatric oncologists participate in the National Pediatric Cancer Foundation Sunshine Project, a collaborative network of over 30 leading U.S. children’s hospitals. This network aims to develop more effective and less toxic treatments for childhood cancer through research, clinical trials and strategic partnerships with academic, industry and global institutions.
Early Drug Development in Pediatric Cancer
Our team has created a comprehensive Early Drug Development Program for patients who don’t have good treatment options or haven’t responded to standard therapy. Phase I and II trials represent the early development of some of the most promising agents.
These trials give our patients access to the newest treatment options without traveling to other centers in Arizona or another state. Phoenix Children’s is the only center in Arizona, New Mexico and Nevada that offers Phase I pediatric oncology trials.
Innovative Preclinical Research
The Pediatric Cancer Immunology and Immunotherapy Lab, led by Principal Investigator Mario Otto, MD, PhD, focuses on developing cell-based, targeted therapies for pediatric cancer. The lab's translational research encompasses graft engineering for hematopoietic stem cell transplantation, nanotechnology, and the development of synthetic lymphoid organoids to enhance immune function in immunocompromised patients. As a bench-to-bedside research lab, it aims to evaluate the synergy and potency of combination immunotherapies and to identify the molecular mechanisms underlying their effects. Additionally, the lab seeks to develop more accurate preclinical models for stem cell transplantation and cancer immunotherapies, leading to clinical trials for patients requiring life-saving hematopoietic stem cell transplantation and other targeted anti-cancer treatments.
Alexandra M. Walsh, MD, interim division director of the leukemia/lymphoma team, medical director of the Childhood Cancer Survivor and Fertility Preservation Programs and associate professor of child health at the University of Arizona College of Medicine, Center for Cancer & Blood Disorders, leads progressive translational research. Her research interests encompass areas of cancer care that extend beyond standard treatment protocols. This includes fertility preservation techniques, supportive care issues – such as improving methods to decrease nausea and vomiting during chemotherapy, as well as strategies to reduce fungal infections – and survivorship care. Dr. Walsh uses a database of 1,100 patients in survivorship to address questions regarding the side effects patients experience after cancer treatment and how to mitigate them. A current project focuses on sending daily text messages to acute lymphoblastic leukemia patients during the intensive phases of their therapy, aiming to reduce hospital readmission rates.
Tanya Kalin, MD, PhD, heads an innovative research lab investigating novel therapies for cancer and interstitial lung diseases. The team engages in drug development, screening therapeutic compounds to inhibit cancer formation and address long-term complications from unresolved lung injuries caused by radiation, chemotherapy or viral infections. By utilizing clinical tissue samples, human ex vivo models and animal models, the lab seeks to unravel the complex signaling pathways involved in disease progression. The team aims to target these abnormalities to enhance patient survival and improve outcomes.
Nawal Merjaneh, MD, a clinician-scientist, is conducting research focused on identifying novel molecular targets and translating these discoveries into targeted therapies for patients with rhabdomyosarcoma. Her current work emphasizes the role of the calcium signaling pathway in both rhabdomyosarcoma cell lines and mouse models. Her research previously received support from CancerFree Kids. She recently received the Young Investigator Award from Hyundai Hope on Wheels, which is instrumental in advancing her ongoing projects.
Ross Mangum, MD, is a leading clinician-scientist specializing in pediatric neuro-oncology. He focuses on developing minimally invasive methods for diagnosing and monitoring brain and spinal cord tumors in children. Dr. Mangum’s work addresses the limitations of traditional tissue biopsies by investigating liquid biopsy techniques to detect circulating tumor DNA (ctDNA) from cerebrospinal fluid (CSF) and blood samples. Recognizing the challenges posed by the blood-brain barrier, Dr. Mangum’s research prioritizes CSF as a source of tumor-specific genetic information, using advanced digital sequencing technologies to track molecular changes over the course of treatment. His goal is to advance diagnostic and surveillance strategies that reduce invasive procedures, ultimately improving care for pediatric patients with brain tumors.
Mona Nourani, DO, an established clinician-scientist, studies the role of extracellular vesicles (EVs) in chemoresistant pediatric osteosarcoma (OS) to identify biomarkers that signal drug resistance mechanisms. Proteomic analysis of OS-derived EVs from drug-resistant preclinical models revealed key proteins, such as TNIK and DDAH1, involved in epithelial to mesenchymal transition (EMT) and the Wnt signaling pathway, suggesting their role in OS chemoresistance. The next phase involves clinical validation of these biomarkers through the analysis of plasma samples from osteosarcoma patients.
Key Local Clinical and Research Partners
Phoenix Children’s collaborates with several key institutions across Arizona, forming a strong network of partnerships dedicated to advancing pediatric healthcare and research.
- The University of Arizona College of Medicine – Phoenix works closely with the hospital to develop educational programs and translational research that push the boundaries of pediatric medicine.
- The Translational Genomics Research Institute (TGen) brings expertise in genomics, offering collaborative opportunities in precision medicine and pediatric cancer research.
- Arizona State University (ASU) provides multidisciplinary collaboration in fields like biomedical engineering and digital health.
- Phoenix Children’s Biorepository is crucial in biospecimen collection and research, supporting innovative clinical trials and studies.
- Mayo Clinic in Arizona partners with Phoenix Children’s on various clinical and research initiatives, enhancing patient care through shared expertise and resources.
These collaborations are pivotal in positioning Phoenix Children’s as a regional leader in pediatric healthcare innovation.
Find a Research Study
Patients and families can use the Find a Research Study tool or speak with their child’s doctor to learn about enrollment in current and upcoming clinical research studies at Phoenix Children’s.